This is an open label, single arm, phase 2 trial investigating bezuclastinib plus sunitinib in patients with GIST who have previously progressed on sunitinib.

开始日期2024-08-01

申办/合作机构

Sarcoma Alliance for Research through Collaboration

[+3]

NCT05186753

/ Active, not recruiting临床2期

A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis

This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM), including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be conducted in three parts. Patients in Parts 1a, 1b and 2 will receive bezuclastinib or placebo, and may roll over onto Part 3 to receive treatment with bezuclastinib. Additionally, a substudy of subjects will investigate the efficacy, safety, and tolerability of bezuclastinib in patients who are experiencing inadequate symptom control with avapritinib.

开始日期2022-06-27

申办/合作机构

Cogent Biosciences, Inc.

NCT05208047

/ Recruiting临床3期

A Phase 3 Randomized, Open-Label, Multicenter Clinical Study of CGT9486+Sunitinib vs. Sunitinib in Subjects With Locally Advanced, Unresectable, or Metastatic Gastrointestinal Stromal Tumors

This is a Phase 3, open-label, international, multicenter study of CGT9486 in combination with sunitinib. This is a multi-part study that will enroll approximately 482 patients. Part 1 consists of two evaluations: 1) confirming the dose of an updated formulation of CGT9486 to be used in subsequent parts in approximately 20 patients who have received at least one prior line of therapy for Gastrointestinal Stromal Tumors (GIST) and 2) evaluating the potential for drug-drug interactions between CGT9486 and sunitinib in approximately 18 patients who have received at least two prior tyrosine kinase inhibitors (TKIs) for GISTs. The second part of the study will enroll approximately 388 patients who are intolerant to, or who failed prior treatment with imatinib only and will compare the efficacy of CGT9486 plus sunitinib to sunitinib alone with patients being randomized in a 1:1 manner. This study also contains two substudies: 1) a drug-drug interactions (DDI) substudy will investigate the potential for CGT9486 to be a Cytochrome P450 (CYP)3A4 inducer in approximately 16 patients who have received at least one prior line of therapy for GIST and 2) a substudy intended to test the efficacy of bezuclastinib and sunitinib as first-line (1L) treatment of GIST in approximately 40 participants with KIT exon 9 mutations and no prior systemic therapy (with the exception of up to 10 subjects with ongoing imatinib therapy of ≤4 weeks).

开始日期2022-04-14

申办/合作机构

Cogent Biosciences, Inc.

100 项与贝祖克拉替尼相关的临床结果

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100 项与贝祖克拉替尼相关的转化医学

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100 项与贝祖克拉替尼相关的专利（医药）

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项与贝祖克拉替尼相关的文献（医药）

2026-04-01·JOURNAL OF GASTROENTEROLOGY AND HEPATOLOGY

Gastrointestinal Stromal Tumors: Molecular Mechanisms of Drug Resistance and Advances in Therapeutic Strategies

Review

作者: Zhi, Ding ; Fazhi, Zhao ; Huachuan, Zhang ; Yan, Chen ; Jingnuo, Ding ; Da, Zhou

ABSTRACT:

Gastrointestinal stromal tumors (GISTs), the most common mesenchymal neoplasms of the digestive tract, are primarily driven by gain‐of‐function mutations in the receptor tyrosine kinases KIT or PDGFRA. The introduction of imatinib, a selective tyrosine kinase inhibitor (TKI), revolutionized the management of advanced GIST. However, the emergence of drug resistance remains a major clinical challenge, limiting long‐term efficacy. Resistance mechanisms are multifactorial, predominantly involving secondary mutations in KIT or PDGFRA that impair TKI binding or stabilize the kinase in an active conformation. Beyond genomic alterations, nonmutational adaptive responses—including dysregulation of noncoding RNAs, metabolic reprogramming, epigenetic modifications, and an immunosuppressive tumor microenvironment—significantly contribute to treatment failure. This review comprehensively analyzes these resistance pathways and discusses the evolution of therapeutic strategies toward precision medicine. Current approaches emphasize molecular subtype‐guided first‐line therapy, ctDNA‐driven sequencing of subsequent lines, and the use of novel TKIs (e.g., avapritinib, ripretinib, bezuclastinib) tailored to specific resistance mutations. Furthermore, we explore emerging modalities such as boron neutron capture therapy (BNCT), which offers a kinase‐independent mechanism to target resistant disease, and combination strategies that integrate immunotherapy, epigenetic modulators, and pathway‐specific inhibitors. Advances in liquid biopsy and molecular profiling are enabling real‐time adaptation of therapy, moving GIST management toward a dynamic, personalized paradigm aimed at overcoming resistance and improving patient outcomes.

2023-07-03·Expert opinion on emerging drugs

Systemic mastocytosis: 2023 update on diagnosis and management in adults

Review

作者: Costa, Alessandro ; Masucci, Chiara ; Carmosino, Ida ; Ielo, Claudia ; Capriata, Marcello ; Passucci, Mauro ; Scalzulli, Emilia ; Martelli, Maurizio ; Breccia, Massimo

INTRODUCTION:

Systemic mastocytosis (SM) is a complex and heterogeneous disease, characterized by the clonal accumulation of mast cells in one or more organs. In 2022 both the World Health Organization (WHO) and the International Consensus Classification (ICC) modified the diagnostic and classification criteria of SM. Moreover, the identification of new clinical and molecular variables has improved prognostic tools and led to increasingly individualized therapeutic strategies.

AREAS COVERED:

The aim of this review is to present the updates introduced by the International Consensus Classification in diagnostic criteria of SM. In addition, we report the latest data available from the most important clinical trials in patients both with non-advanced and advanced disease, including elenestinib and bezuclastinib.

EXPERT OPINION:

Diagnosis and classification of SM has evolved over years. The most recent WHO and ICC classification improved SM diagnostic work-up, providing clinicians with a clear and simplified diagnostic scheme. New approved targeted therapies such as midostaurin and avapritinib modified the treatment paradigm in patients in advanced stage, and next-generation inhibitors actually investigated in clinical trials are expected in the next future.

2022-12-09·Hematology. American Society of Hematology. Education Program

Available and emerging therapies for bona fide advanced systemic mastocytosis and primary eosinophilic neoplasms

Review

作者: Gotlib, Jason

Abstract:

The historically poor prognosis of patients with advanced systemic mastocytosis (AdvSM) and primary eosinophilic neoplasms has shifted to increasingly favorable outcomes with the discovery of druggable targets. The multikinase/KIT inhibitor midostaurin and the highly selective KIT D816V inhibitor avapritinib can elicit marked improvements in measures of mast cell (MC) burden as well as reversion of MC-mediated organ damage (C-findings) and disease symptoms. With avapritinib, the achievement of molecular remission of KIT D816V and improved survival compared with historical therapy suggests a potential to affect disease natural history. BLU-263 and bezuclastinib are KIT D816V inhibitors currently being tested in trials of AdvSM. In the new World Health Organization and International Consensus Classifications, the category of “myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase (TK) gene fusions” is inclusive of rearrangements involving PDGFRA, PDGFRB, FGFR1, JAK2, FLT3, and ETV6::ABL1. While the successful outcomes with imatinib in FIP1L1::PDGFRA-positive cases and PDGFRB-rearranged neoplasms have become the “poster children” of these disorders, the responses of the other TK-driven neoplasms to small-molecule inhibitors are more variable. The selective FGFR inhibitor pemigatinib, approved in August 2022, is a promising therapy in aggressive FGFR1-driven diseases and highlights the role of such agents in bridging patients to allogeneic transplantation. This review summarizes the data for these approved and investigational agents and discusses open questions and future priorities regarding the management of these rare diseases.

177

项与贝祖克拉替尼相关的新闻（医药）

2026-07-02

·世易医健

ASCO 2026开启胰腺癌新时代，靶向治疗版图扩展至整个消化道肿瘤领域

在2026年美国临床肿瘤学会（ASCO）年会上，多位肿瘤领域专家接受了采访并分享了对于消化系统肿瘤及胃肠间质瘤（GIST）领域潜在“改变临床实践”研究的看法。这些研究成果有望在未来逐步转化为临床实践。以下为专家观点与重点研究内容。胰腺癌 RASolute 302研究：Daraxonrasib对比化疗用于既往治疗后转移性胰腺导管腺癌（摘要LBA5）毫无疑问，今年ASCO最受关注的研究之一来自大会全体会议。Ⅲ期RASolute 302研究（NCT06625320）结果显示，每日一次口服RAS(ON)多选择性抑制剂daraxonrasib（n=248），相比化疗（n=252），在既往接受治疗的转移性胰腺导管腺癌患者中显著改善总生存期（OS）和无进展生存期（PFS）。在RAS G12突变患者中，daraxonrasib组（n=228）中位OS为13.2个月（95% CI，10.0-未达到[NE]），而化疗组（n=231）仅为6.6个月（95% CI，5.4-8.2），死亡风险降低60%（HR，0.40；95% CI，0.30-0.54；P＜0.0001）。12个月OS率分别为53.3%和18.7%。中位PFS分别为7.3个月（95% CI，6.3-8.1）和3.5个月（95% CI，2.9-3.8）（HR，0.45；95% CI，0.34-0.59；P＜0.0001）。在意向治疗（ITT）人群中，结果同样一致：daraxonrasib组中位OS为13.2个月，而化疗组为6.7个月（HR，0.40；P＜0.0001）；中位PFS分别为7.2个月和3.6个月（HR，0.49；P＜5.2×10⁻⁸）。此外，与化疗相比，daraxonrasib还延缓了患者疼痛恶化及总体生活质量下降。其安全性可控，未发现新的安全信号。截至数据截止时，daraxonrasib组仍有42%的患者持续接受治疗，而化疗组仅为14%。 Vincent Picozzi教授美国弗吉尼亚Mason 医学中心 “RASolute 302研究的公布，是自化疗出现以来胰腺癌领域最具里程碑意义的进展……这真正开启了胰腺癌治疗的新篇章。我把它称为一次‘彻底重来’，因为我们如今拥有了新的治疗基础。过去对于胰腺癌的很多认知和判断都需要被重新审视，甚至被改变。我认为，这类药物未来几乎会影响每一位胰腺癌患者的治疗。” Benjamin Herzberg教授美国哥伦比亚大学Herbert Irving综合癌症中心 “在一种长期缺乏有效治疗选择的疾病中，实现OS翻倍，本身已经说明了很多问题。首先，它证明了只要命中正确靶点，药物就会有效。其次，它也证明KRAS不仅仅是G12C可以被靶向，如今借助‘分子胶’等新型技术平台，KRAS本身已经成为可成药靶点。这项研究实际上为未来多种KRAS靶向策略以及肿瘤靶向治疗提供了概念验证。” Ferdinandos Skoulidis教授美国MD Anderson癌症中心 “RASolute 302无疑是今年ASCO最重磅、最具影响力的研究。它在大会全体会议上获得全场起立鼓掌，这足以说明其意义。作为一名曾经在胰腺癌和KRAS领域开始科研与临床训练、后来转向肺癌研究的医生，我认为能够看到一种靶向治疗将胰腺癌患者中位OS提高超过一倍，是历史性的时刻。” Kevin Kalinsky教授美国埃默里大学Winship癌症研究所 “RAS一直是我们多年来希望攻克的靶点，但长期以来都被认为‘不可成药’。如今终于有药物能够成功靶向这一突变，本身已经非常令人振奋。更重要的是，胰腺癌一直是一个极难取得突破的瘤种，因此看到这一领域终于出现重大进展，极具鼓舞意义。这也预示着未来其他RAS突变肿瘤的药物研发方向。” Daniel King教授美国秒佑医疗国际 “虽然这可能是一项突破性进展，但还不能称之为‘奇迹’，因为它并不是治愈。接下来我们必须解决的问题是：患者为何会对RAS靶向治疗产生耐药？我们如何通过联合更多RAS抑制策略，或更聪明地设计针对耐药机制的临床研究来克服耐药？还有大量工作需要完成，但这无疑是令人兴奋的结果。” Robert Vonderheide教授美国宾夕法尼亚大学Abramson癌症中心 “尽管daraxonrasib令人兴奋，但它只是胰腺癌整体进展背景中的一部分。未来不会只有一种药物，而是会有多种治疗选择，在合适的时间匹配合适的患者。” Shubham Pant教授美国MD Anderson癌症中心 “RASolute 302说明，今年就是属于胰腺癌的一年。我认为ASCO历史上从未真正有过‘属于胰腺癌的一年’，直到现在。” 结直肠癌 BREAKWATER研究：Encorafenib + Cetuximab + FOLFIRI用于BRAF V600E突变转移性结直肠癌一线治疗（摘要LBA3503） Ⅲ期BREAKWATER研究（NCT04607421）队列3最终PFS及更新OS数据显示，在既往未经治疗的BRAF V600E突变转移性结直肠癌患者中，encorafenib联合西妥昔单抗（cetuximab）及FOLFIRI方案，相较FOLFIRI±贝伐珠单抗，几乎使PFS翻倍。试验组中位PFS达到15.2个月（95% CI，13.6-NE），对照组为8.6个月（95% CI，6.8-8.9）（HR，0.44；P=0.001）。中位随访20.6个月时，试验组中位OS尚未达到，而化疗组中位OS为20.3个月（HR，0.56）。18个月OS率分别为72.0%和54.9%。该联合方案安全性与各组成药物既往已知特征一致，未观察到新的安全信号。 Paul Oberstein教授美国纽约大学Langone Perlmutter癌症中心 “BREAKWATER是今年ASCO最重要、最具影响力的研究之一。BRAF突变结直肠癌一直非常难治，而该研究中的新型联合方案在这一患者群体中显示出显著获益。结合本届ASCO其他研究结果，BRAF突变结直肠癌患者的靶向治疗机会正在不断扩大。” 胃食管癌 HERIZON-GEA-01研究：Zanidatamab联合化疗±替雷利珠单抗用于HER2阳性局部晚期或转移性胃食管腺癌一线治疗（摘要4010） Ⅲ期HERIZON-GEA-01研究（NCT05152147）预设PD-L1亚组分析显示，无论PD-L1表达状态如何，包括PD-L1阴性患者，zanidatamab-hrii联合替雷利珠单抗（tislelizumab）及化疗，相较曲妥珠单抗（trastuzumab）联合化疗，均能改善HER2阳性局部晚期或转移性胃食管腺癌患者的PFS和OS。此前公布的主要结果显示，联合方案组（n=302）中位PFS为12.4个月，而曲妥珠单抗组（n=308）为8.1个月（HR，0.63；P＜0.0001）；中位OS分别为26.4个月和19.2个月（HR，0.72；P=0.0043）。此次ASCO公布的PD-L1亚组分析进一步证实，无论采用TAP评分还是CPS评分评估PD-L1状态，PFS改善均保持一致。值得注意的是，在PD-L1阴性（TAP＜1%）患者中，联合方案组中位OS达到29.7个月，而曲妥珠单抗组仅为15.8个月。 Geoffrey Y. Ku教授美国纪念斯隆凯特琳癌症中心 “这些数据再次验证了此前研究公布及发表结果所显示的趋势：无论PD-L1阴性还是阳性患者，似乎都能从zanidatamab联合替雷利珠单抗及化疗方案中获益。虽然目前我们仍不清楚其背后的分子机制，但希望未来通过进一步相关性分析能够逐步揭示原因。” 胃肠间质瘤（GIST） PEAK研究：Bezuclastinib联合舒尼替尼对比舒尼替尼单药用于晚期GIST（摘要11500） Ⅲ期PEAK研究（NCT05208047）主要结果显示，在伊马替尼耐药或不耐受的晚期GIST患者中，bezuclastinib联合舒尼替尼（sunitinib）可将疾病进展或死亡风险降低50%，且客观缓解率（ORR）几乎翻倍。这也是该领域首个在对照活性药物基础上显示统计学显著优势的治疗方案。截至2025年9月30日数据截止时，联合方案组中位PFS为16.5个月（95% CI，13.8-19.2），而舒尼替尼单药组仅为9.2个月（95% CI，7.2-11.0）（HR，0.50；P＜0.0001）。值得关注的是，无论KIT原发或继发突变状态如何，联合方案均表现出一致的PFS获益，提示其在二线GIST复杂异质性突变背景下具有广泛活性。安全性方面，该联合方案总体耐受性良好，未观察到超出舒尼替尼既往已知范围的新安全风险。 Michael Heinrich教授美国俄勒冈健康与科学大学Knight癌症研究所 “我对PEAK研究结果非常兴奋。这是一项阳性的Ⅲ期研究，中位PFS从9个月延长至16个月，而联合治疗并未显著增加毒性，这在二线治疗中是前所未有的。” Andrew Wagner教授美国Dana-Farber癌症研究所/哈佛医学院 “今年肉瘤领域其实有不少重要研究，其中PEAK研究尤为突出。Bezuclastinib联合舒尼替尼有望改变临床实践，并取代舒尼替尼单药成为GIST患者二线治疗新标准。”

2026-06-30

·药明康德

抗癌双特异性抗体达到3期临床主要终点；创新小分子疗法向美国FDA提交新药申请…… | 行业新闻

治疗弥漫性大B细胞淋巴瘤，双特异性抗体达到3期临床主要终点艾伯维（AbbVie）和Genmab日前公布了3期临床试验EPCORE DLBCL-4的顶线结果。该试验在既往接受过至少一线治疗的复发或难治性（R/R）弥漫性大B细胞淋巴瘤（DLBCL）成人患者中，评估了T细胞衔接双特异性抗体epcoritamab联合来那度胺，相较于利妥昔单抗联合吉西他滨和奥沙利铂（R-GemOx）的疗效。根据预设主要分析的顶线结果，该试验达到主要终点，显示出具有统计学显著性和临床意义的无进展生存期（PFS）改善。根据美国和美国以外地区不同的删失规则，疾病进展或死亡风险分别降低60%（HR=0.40；95% CI：0.30，0.55；p<0.0001）和56%（HR=0.44；95% CI：0.33，0.60；p<0.0001）。 Epcoritamab是一款采用Genmab专有DuoBody技术开发的IgG1双特异性抗体，通过皮下注射给药。Genmab的DuoBody-CD3技术旨在选择性地引导细胞毒性T细胞，诱导其针对靶细胞类型产生免疫反应。Epcoritamab可同时结合T细胞上的CD3和B细胞上的CD20，并诱导T细胞杀伤CD20阳性细胞。创新小分子疗法向美国FDA提交新药申请 Cogent Biosciences今日宣布，已向美国FDA提交bezuclastinib用于治疗晚期系统性肥大细胞增多症（AdvSM）的新药申请（NDA）。此次申请基于关键性APEX试验取得的积极临床数据。相关数据最近在2026年欧洲血液学协会（EHA）大会上公布。截至2026年3月31日数据截止时，共有81例AdvSM患者接受bezuclastinib（150 mg）治疗。在68例疗效可评估患者中，根据修订版国际骨髓增殖性肿瘤研究与治疗工作组-欧洲肥大细胞增多症能力网络（mIWG-MRT-ECNM）标准评估的主要终点缓解情况显示，总缓解率（ORR）为65%。此外，bezuclastinib展现出持久的临床活性和较长的无进展生存期（PFS），12个月无进展生存率为79%，12个月总生存率（OS）为87%。 Bezuclastinib是一种选择性酪氨酸激酶抑制剂，旨在强效抑制关键KIT驱动突变所产生的突变蛋白活性。其中KIT D816V突变会驱动系统性肥大细胞增多症，这是一种由肥大细胞失控增殖引起的严重疾病。赛诺菲庞贝病疗法达到3期临床主要终点赛诺菲（Sanofi）今日宣布，Nexviazyme（avalglucosidase alfa）在3期临床试验Baby-COMET中取得积极结果。Nexviazyme达到主要终点，Nexviazyme达到主要终点，即既往未接受治疗、年龄在6个月及以下的婴儿期起病庞贝病（IOPD）儿科受试者在接受治疗52周时存活且无需有创通气的比例。此外，该研究达到所有次要终点，包括受试者在12个月和18个月龄时存活且无需有创通气的比例，以及治疗52周时其他疾病进展指标的数值改善。这些数据将支持在美国提交标签扩展申请，预计提交时间为2026年下半年。庞贝病是一种罕见的遗传性、进展性神经肌肉疾病，由酸性α-葡萄糖苷酶（GAA）缺乏引起，会影响全身肌肉功能。Nexviazyme是一种酶替代疗法，正被评估作为IOPD的一种潜在治疗选择。在Baby-COMET研究中，Nexviazyme耐受性良好，安全性与其既往的安全性特征一致；未发生严重的治疗相关不良事件、死亡或停药事件，29.4%的受试者出现可控的输注相关反应。参考资料： [1] AbbVie Announces Positive Phase 3 Results for Epcoritamab Plus Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma. Retrieved June 30, 2026, from https://www.prnewswire.com/news-releases/abbvie-announces-positive-phase-3-results-for-epcoritamab-plus-lenalidomide-in-patients-with-relapsed-or-refractory-diffuse-large-b-cell-lymphoma-302813689.html [2] Cogent Biosciences Announces Submission of New Drug Application (NDA) for Bezuclastinib in Advanced Systemic Mastocytosis (AdvSM). Retrieved June 30, 2026, from https://investors.cogentbio.com/news-releases/news-release-details/cogent-biosciences-announces-submission-new-drug-application-nda [3] Press Release: Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study. Retrieved June 30, 2026, from https://www.globenewswire.com/news-release/2026/06/30/3319382/0/en/press-release-sanofi-s-nexviazyme-met-all-primary-and-secondary-endpoints-in-infantile-onset-pompe-disease-phase-3-study.html 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新……

临床3期临床结果临床2期临床1期

2026-07-06

·BioSpace

Cogent Biosciences Announces Submission of New Drug Application (NDA) for Bezuclastinib in Advanced Systemic Mastocytosis (AdvSM)

Submission based on results of APEX pivotal trial which demonstrated bezuclastinib objective response rate (CR+CRh+PR+CI) of 65% per mIWG criteria and 81% ORR per PPR criteria with a well-tolerated safety pro AdvSM patients APEX NDA is third submission for bezuclastinib in six months; PEAK and SUMMIT reviews ongoing and on track for anticipated Q4 2026 approval WALTHAM, Mass. and BOULDER, Colo., June 30, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced it has submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM). The submission is based on positive clinical data from the APEX pivotal trial. “We are pleased to announce the submission of our NDA for patients with Advanced Systemic Mastocytosis based on the impressive results from the APEX trial which demonstrated that a highly selective KIT mutant inhibitor like bezuclastinib can deliver clear clinical benefit without the severe tolerability challenges associated with currently available treatments,” said Andrew Robbins, President and Chief Executive Officer. “We are working closely with the FDA on all three of our submissions and remain confident that bezuclastinib will achieve its first approvals later this year. I’d also like to congratulate our small, but exceptionally talented, Cogent development team, whose amazing work analyzing, presenting and submitting data from three pivotal trials in less than twelve months has rapidly positioned the company to achieve our vision of creating best-in-class therapies for patients fighting rare, mutational driven diseases.” The NDA submission is supported by data from the pivotal APEX trial, which were most recently presented at the 2026 European Hematology Association (EHA) Congress. As of the March 31, 2026 data cutoff, 81 AdvSM patients were treated with 150 mg of bezuclastinib, including 57 patients with SM-AHN, 11 patients with ASM and 13 patients with MCL. The primary endpoint of response per mIWG-MRT-ECNM was assessed on 68 evaluable patients and showed 65% ORR (CR+CRh+PR+CI), including 57% of patients who achieved CR, CRh or PR as best response. Additional highlights included: Key secondary endpoint of response per pure pathological response (PPR) criteria was assessed on 81 patients which showed an 81% ORR (CR+CRh+PR). Bezuclastinib demonstrated reversal of bone marrow pathobiology including rapid and deep reductions in aberrant CD25 and CD30 expression, normalization of mast cell morphology, normalization of bone marrow cellularity, and improvement in myelofibrosis. Bezuclastinib demonstrated durable clinical activity and prolonged PFS with a 12-month PFS rate of 79% and a 12-month OS rate of 87%. Median duration of PFS and OS were immature at the time of the data cutoff. Bezuclastinib achieved clear and clinically significant reductions in objective disease markers for these AdvSM patients: Outcome measure Bezuclastinib Proportion with ≥50% reduction in serum tryptase (n=80) 89 % Proportion with ≥50% reduction in bone marrow mast cells or clearance of aggregates (n=80) 89 % Proportion with ≥50% reduction in KIT D816V variant allele frequency (n=43) 91 % In the APEX study, bezuclastinib also demonstrated robust improvement in disease pathology, with effects observed as early as eight weeks, including high PPR rates, improvement (including normalization) in bone marrow mast cell distribution, improvement in broader bone marrow characteristics and a majority of patients achieving normalization of serum tryptase. In addition, approximately one-third of patients treated with bezuclastinib achieved undetectable levels of KIT D816V VAF, suggesting modification of the underlying AdvSM disease with bezuclastinib treatment. As of the data cutoff, bezuclastinib was well-tolerated, with infrequent need for dose reduction or discontinuation for treatment-related adverse events (TRAEs). The most frequent TRAEs reported on bezuclastinib treatment were hair color change (31%), neutropenia (31%), altered taste (28%), thrombocytopenia (25%), and ALT/AST elevations (21%). The majority of transaminase elevations were of low grade, asymptomatic and reversible. Of the two patients who experienced Grade 3 transaminase elevation, one discontinued treatment and one remains on therapy following dose reduction. Bezuclastinib - Expanded Access Program Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients SM or GIST who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. For more information please visit: . Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) Cogent also announced today that, on June 26, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to two new employees under the company’s 2020 Inducement Plan with a grant date of June 29, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 7,600 shares of Cogent common stock and (ii) 7,000 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date. About Cogent Biosciences, Inc. Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Kα, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at  . Follow Cogent Biosciences on social media:  X  (formerly known as Twitter) and  LinkedIn . Information that may be important to investors will be routinely posted on our website and  X . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the expectation that both of the company’s NDAs for bezuclastinib in GIST and NonAdvSM will be approved by the FDA in the fourth quarter of 2026 and the expectation that the company will achieve its vision of creating best-in-class therapies for patients fighting rare, mutational driven diseases. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Annual Report on Form 10-K, as supplemented by Quarterly Reports on Form 10-Q and other filings Cogent makes with the SEC from time to time . Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. Contact: Christi Waarich Senior Director, Investor Relations 617-830-1653 CONTACT: christi.waarich@cogentbio.com

临床结果申请上市临床研究

100 项与贝祖克拉替尼相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
侵袭性系统性肥大细胞增多症	申请上市	美国	Cogent Biosciences, Inc.	2026-06-30
胃肠道间质瘤	申请上市	美国	Cogent Biosciences, Inc.	2026-04-01
转移性胃肠道间质瘤	临床3期	美国	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	阿根廷	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	澳大利亚	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	巴西	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	加拿大	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	智利	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	捷克	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	丹麦	Cogent Biosciences, Inc.	2022-04-14

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05208047 (Peak, ASCO2026)	临床3期	胃肠道间质瘤二线	413	Bezuclastinib 600 mg QD + Sunitinib 37.5 mg QD	廠齋顧憲壓簾壓鑰築顧(鏇遞鑰網鏇窪鹹遞築壓) = 憲糧夢願積範廠憲範鏇淵簾網衊範網齋壓衊構 (鏇繭窪餘範窪淵鏇膚夢, 13.8 ~ 19.2) 更多	积极	2026-05-29
				Sunitinib 37.5 mg QD	廠齋顧憲壓簾壓鑰築顧(鏇遞鑰網鏇窪鹹遞築壓) = 餘遞鬱壓選構網願鑰範淵簾網衊範網齋壓衊構 (鏇繭窪餘範窪淵鏇膚夢, 7.2 ~ 11.0) 更多
NCT04996875 (Apex, EHA2026)	临床2期	侵袭性系统性肥大细胞增多症 KIT D816V	81	Bezuclastinib 150 mg once daily	蓋鬱簾鏇製繭淵築壓範(積衊憲壓願顧壓糧願壓) = 糧鏇糧積鏇鹽鹹蓋醖繭齋窪蓋簾簾鏇艱鹽獵廠 (醖鏇窪願蓋繭觸鑰築製 ) 更多	积极	2026-05-12
NCT05186753 (Summit, AAAAI2026)	临床2期	系统性肥大细胞增多症 KIT c.2447 C>T (p.D816V) mutation	12	Bezuclastinib	衊構淵範衊遞網膚獵構(夢獵網鏇鏇鑰糧獵簾壓) = 願築膚齋範艱膚餘艱憲網鏇窪選觸製築蓋鏇繭 (鹽膚襯築齋夢獵願鬱鹹 ) 更多	积极	2026-02-27
NCT05186753 (Summit, AAAAI2026)	临床2期	系统性肥大细胞增多症 KIT c.2447 C>T (p.D816V) mutations	179	Bezuclastinib	襯鬱膚窪遞選膚觸鹽憲(窪艱顧遞醖襯糧鹹蓋遞) = 構壓鏇艱鬱繭鑰壓餘觸網網糧鬱築構簾糧壓鹽 (齋構廠憲襯繭齋醖遞蓋 ) 更多	积极	2026-02-27
				Placebo	襯鬱膚窪遞選膚觸鹽憲(窪艱顧遞醖襯糧鹹蓋遞) = 範淵蓋遞壓廠顧艱選夢網網糧鬱築構簾糧壓鹽 (齋構廠憲襯繭齋醖遞蓋 ) 更多
NCT05186753 (Summit, AAAAI2026)	临床2期	系统性肥大细胞增多症 KIT c.2447 C>T (p.D816V)	179	Bezuclastinib	遞選顧願遞獵願觸醖鑰(艱遞淵遞鹽夢鑰簾窪餘) = 襯鑰窪膚製繭觸網壓襯衊選簾蓋積觸齋鹽廠壓 (鑰選夢膚壓膚壓糧壓艱 )	积极	2026-02-27
				Placebo	遞選顧願遞獵願觸醖鑰(艱遞淵遞鹽夢鑰簾窪餘) = 鏇蓋願鹽獵淵窪鏇窪廠衊選簾蓋積觸齋鹽廠壓 (鑰選夢膚壓膚壓糧壓艱 )
NCT05186753 (Summit, ASH2025) 人工标引	临床2期	系统性肥大细胞增多症 KIT c.2447 C>T (p.D816V) mutation	179	Bezuclastinib + BSC	壓鹹鏇網繭鹹願製構蓋(餘製糧餘遞觸鹽廠醖鹹) = 淵憲鑰窪網淵糧選觸築餘鬱襯願夢淵醖簾鏇壓 (築選積顧廠蓋鹽願憲積, -27.6 ~ -21.1) 更多	积极	2025-12-06
				Placebo + BSC	壓鹹鏇網繭鹹願製構蓋(餘製糧餘遞觸鹽廠醖鹹) = 構襯糧鹽醖遞糧壓壓壓餘鬱襯願夢淵醖簾鏇壓 (築選積顧廠蓋鹽願憲積, -19.6 ~ -11.2) 更多
NCT05208047 (PEAK, Company_Website) 人工标引	临床3期	胃肠道间质瘤二线	-	bezuclastinib + sunitinib	窪鹹遞艱鹹淵廠鹹鬱衊(獵壓鹽憲糧糧夢淵獵鑰) = 窪顧願繭艱鑰膚窪鏇製選簾齋衊鑰構簾醖艱壓 (製鏇積鬱廠鹹齋衊網選 ) 更多	积极	2025-11-10
				sunitinib	窪鹹遞艱鹹淵廠鹹鬱衊(獵壓鹽憲糧糧夢淵獵鑰) = 蓋構簾衊鏇願願蓋衊範選簾齋衊鑰構簾醖艱壓 (製鏇積鬱廠鹹齋衊網選 ) 更多
NCT05186753 (SUMMIT, Corporate Publications 1 \| Corporate Publications 2 \| Company_Website) 人工标引	临床2期	系统性肥大细胞增多症 \| 肥大细胞增多症	179	Bezuclastinib	範積蓋憲積積選憲膚壓(積觸簾願醖膚鹽醖齋鹹) = 淵鬱鬱鏇簾襯齋構窪糧鹽醖艱夢顧顧顧鏇繭艱 (遞範餘憲願廠鑰壓衊獵 ) 达到更多	积极	2025-07-07
				placebo	範積蓋憲積積選憲膚壓(積觸簾願醖膚鹽醖齋鹹) = 餘壓廠糧築鏇製壓選齋鹽醖艱夢顧顧顧鏇繭艱 (遞範餘憲願廠鑰壓衊獵 ) 达到更多
NCT06208748 (SARC044, ASCO2025)	临床2期	胃肠道间质瘤 KIT exon 9	40	Bezuclastinib 600 mg daily + Sunitinib 37.5 mg daily	遞鬱艱窪願廠襯選夢醖(窪繭簾艱窪願鹽選鹽衊) = 選鏇積襯簾鹹窪觸艱觸鬱網窪構膚繭積衊淵淵 (鏇鏇壓壓鹽壓膚鹽鹹獵 )	积极	2025-05-30
NCT05186753 (SUMMIT, Company_Website) 人工标引	临床2期	系统性肥大细胞增多症	-	Bezuclastinib 100 mg	鑰憲夢憲範鏇範壓醖鬱(繭憲願膚醖鏇衊網顧齋) = 夢蓋餘糧餘觸範簾願廠憲積壓繭鏇構觸積鏇觸 (襯顧淵鏇蓋構衊製餘鏇 ) 更多	积极	2025-02-27
				Placebo	-